Growth Hormone Use in Adults With Prader-Willi Syndrome

The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months.

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: Nutropin AQ

Detailed Description

The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months. Specific outcomes to be evaluated are as follows:

• Increased IGF-1 as a function of human growth hormone dosage compared with baseline.

1. Improvement of indicators or risk factors for co-morbid diseases [diabetes (by measuring insulin and glucose levels), cardiovascular disease (by measuring lipids and fatty acids), and pulmonary function] in participants.
2. Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants.
3. Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants.
4. Increased energy expenditure as determined by whole-room calorimeter measures (8 hour energy expenditure, RMR, TEF, mechanical work); diet records, physical activity monitors and strength measures.

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • The Children's Mercy Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 60 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 16 to 60 years old
• Male or female with diagnosed Prader-Willi syndrome confirmed by genetic testing (e.g., mPCR)
• Low IGF-1 level (e.g.,≤25%) at baseline
• Ability to provide informed consent or availability of a suitable legally authorized representative

Exclusion Criteria:

• Pregnancy
• Previous treatment with growth hormone
• Uncontrolled endocrine disease, (i.e. diabetes or thyroid)
• History of severe scoliosis
• Heart disease
• Uncontrolled high blood pressure or history of stroke
• Morbid obesity (using PWS growth charts)
• Severe sleep apnea or known breathing difficulties/obstruction (as per history or diagnostic testing results

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Primary Cohort; Nutropin AQ	Drug: Nutropin AQ; 0.0125 mg/kg/day; Other Names: Growth Hormone

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Body Composition	Anthropometric Measures and Body Composition	12 and 24 months
Blood Chemistry	Insulin-like growth factor I- IGF-1	12 and 24 months
Physical Activity	Duration of daily physical activity	12 and 24 months

Collaborators and Investigators

• Sponsor: Children's Mercy Hospital Kansas City
• Investigators: Principal Investigator: Merlin G Butler, MD, Children's Mercy Hospital Kansas City

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2005
• Primary Completion (Actual): January 1, 2012
• Study Completion (Actual): January 1, 2012

Study Registration Dates

• First Submitted: March 6, 2007
• First Submitted That Met QC Criteria: March 7, 2007
• First Posted (Estimate): March 8, 2007

Study Record Updates

• Last Update Posted (Actual): January 8, 2021
• Last Update Submitted That Met QC Criteria: December 15, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormones
• Other Study ID Numbers: 303-C02R